Small molecule therapies that target specific CFTR variants have transformed cystic fibrosis (CF) treatment.
Researchers expressed 43 rare missense CFTR variants in CF bronchial epithelial cells, finding drug response correlated with CFTR function for ivacaftor and lumacaftor therapies.
Most variants demonstrated greater effectiveness with the ivacaftor-lumacaftor combination therapy, suggesting that individuals with CF carrying these variants can potentially benefit significantly from these treatments, especially in combination.
The first total synthesis of Rolloamide B, a cyclic proline-enriched heptapeptide, is reported. This work features solution phase benzotriazole-mediated peptide synthesis ligating native amino acids.